Jakafi Improves Survival and Quality of Life with Myelofibrosis

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Among patients with intermediate- or high-risk myelofibrosis, Jakafi™ (ruxolitinib) reduced spleen size, relieved symptoms, and improved overall survival. The results of this Phase III clinical trial were published in the New England Journal of Medicine.

Myelofibrosis is a chronic disease characterized by abnormal blood cell development and the formation of scar tissue in the bone marrow. Symptoms of myelofibrosis can include fatigue, shortness of breath, abdominal discomfort, pain under the ribs, feeling full, muscle and bone pain, itching, and night sweats. Most patients with myelofibrosis have an enlarged spleen, and many have an enlarged liver.

For people who are experiencing symptoms from myelofibrosis, previous approaches to treatment have included blood transfusions, drug therapy, radiation therapy, surgery to remove the spleen, or stem cell transplantation.

Jakafi was the first drug approved specifically for myelofibrosis. It is taken orally and inhibits two enzymes—JAK1 and JAK2—that contribute to myelofibrosis.

The current study enrolled 309 patients from 89 centers in theUnited States. The patients had intermediate-2 or high-risk myelofibrosis. Half received Jakafi and half received a placebo.

A substantial reduction in the size of the spleen (a 35 percent or greater reduction in volume) occurred in 42 percent of patients in the Jakafi group and less than 1 percent of patients in the placebo group.

A 50 percent or greater reduction in total symptom score was experienced by 46 percent of patients in the Jakafi group and 5 percent of patients in the placebo group.

After roughly one-year, 8 percent of patients in the Jakafi group and 16 percent of patients in the placebo group had died.

Anemia and low platelet counts were more common in the Jakafi group than in the placebo group, but could generally be managed.

Jakafi did not reverse the damage that had been done to the bone marrow by myelofibrosis.

These results demonstrate that Jakafi improves both quality and duration of life among patients with intermediate- or high-risk myelofibrosis. This is an important new treatment option for patients with this condition.